Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy

Recruiting Observational Study
Cardiomyopathy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
0 – 18
Sex
Any
Study type
Observational
Participants needed
200 (estimated)
Sponsor
Tenaya Therapeutics · Industry
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About This Trial

The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.

Trial Locations
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Eligibility Criteria
Retrospective Inclusion Criteria: * Data is available for patient \<18 years of age. Patients must be \<18 years of age at enrollment or at time of death. * Documented results of genotyping showing the presence of at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygo…
Contacts

Matthew Pollman, MD

650-209-8092

mpollman@tenayathera.com

CONTACT

LaTanya Tomlinson

clinical.trials@tenayathera.com

CONTACT